LEONARDO ANDRES SIRULNIK: Yeah, I heard you had a couple of times. Were you sweating a lot tonight?

VINAY CHAKRAVARTHY: Yeah. Yeah.

LEONARDO ANDRES SIRULNIK: Last night?

VINAY CHAKRAVARTHY: Mmhmm.

LEONARDO ANDRES SIRULNIK: Nothing, anywhere, no? Anything new?

VINAY CHAKRAVARTHY: No.

LEONARDO ANDRES SIRULNIK: What about your rash? Can you see this, Kendra?

VINAY CHAKRAVARTHY: Oh, I...

KENDRA CHURCH: Mm-hmm. Yeah, the little white...

VINAY CHAKRAVARTHY: I felt tired. Uh, I was losing weight, but I attributed that just to being a resident and being busy. "I'm not sleeping well, I'm not eating well, you know, I'm working, you know, a lot of hours a week."

VINAY CHAKRAVARTHY: Yeah, I started getting mouth sores, and then I just, I, you know, kind of thought that they were just stress ulcers 'cause I was kind of stressed out at work.

KATIE FILLIPON: You comfortable there? Okay, yeah that's fine...

VINAY CHAKRAVARTHY: Actually, what got me into the emergency room was: I was at home, and I was -- had excruciating back pain.

KATIE FILLIPON: First dose is usually always the worst.

VINAY CHAKRAVARTHY: Uh-huh.

KATIE FILLIPON: Sorry.

VINAY CHAKRAVARTHY: And I called Boston Medical Center's emergency room 'cause, you know, I know the people there, thought I could just kind of get a bed, and the guy was like, "Yeah, yeah, come in." He's like, "I think it's a kidney stone." My labs came back; my white count was out of the roof, my platelets were really low, and initially, I, I - I just couldn't believe it. I saw it and I was like, no way; there's no way. I'm too healthy for that. And then he's like, "Okay, well, let's draw it again," 'cause he thought the same thing. And he drew it again, and the same values came back, and that's when, in my mind, I started thinking leukemia.

KATIE FILLIPON: Any headaches today?

VINAY CHAKRAVARTHY: No.

KATIE FILLIPON: Good. I know that they gave you some Oxycodone last night.

VINAY CHAKRAVARTHY: We have a big L.A. Mega-Drive coming up...

VINAY CHAKRAVARTHY: You know, if you wanna volunteer for bone marrow drives, we kind of s -- step-by-step tell them how they have to do it. India's Independence Day is coming up, I think on August, I think it's like 15th, so we're trying to have a big, like, nationwide independence, kind of, drive, so those are two , two of the big drives that are kind of in the works. It's an issue, for a South Asian to find a match, and it's an issue, I mean, not only for South Asian - say it's my South Asian community - but minority communities. We're under represented, And initially we we started off saying, "Okay, we're going to try to get 20,000 registrants." And then when we got to 20,000, we're like, "Wait a second, why are we stopping here?" Because I'm not the first person to go through this, neither will I be the last person to go through this.

LEONARDO ANDRES SIRULNIK: So he needs at least four weeks from the last chemotherapy to do anything...

KENDRA CHURCH: To recover...

LEONARDO ANDRES SIRULNIK: ...so the idea is that whether he recovers or not, he will go...

KENDRA CHURCH: ...to transplant.

LEONARDO ANDRES SIRULNIK: ...to the transplant.

KATIE FILLIPON: Vinay Chakravarthy, 2-1-4-7-7-8-0-7.

VINAY CHAKRAVARTHY: I'll get hit with chemo and chemo and chemo, over and over again, or the transplant. The first ones are supposed to get you into remission, where you have a less than a certain amount of percentage of leukemia cells in your, in your body. And then they suppress my bone marrow with high-dose chemo for two days, and then they do four days of total body radiation to destroy everything in my bone marrow. Just, just to wipe everything out. Just wipes it all out.

KATIE FILLIPON: This shouldn't take long.

NARRATOR: The conventional treatment for cancer, to blast the entire body, was the approach we heard about growing up; the approach that eventually worked for childhood leukemia. But the Holy Grail in the medical community was a magic bullet that could selectively attack only cancer cells. At the same time my husband and I were in the Boston hospitals, we heard buzz about the first such "smart bomb" in the war on cancer: a pill called Gleevec.

NEWS REPORTER 1: The first announcement Thursday was U.S. marketing approval for the pill called Gleevec...

NEWS REPORTER 2: Cancer specialists are hailing the development of a new drug with remarkable results.

GEORGE DEMETRI: I don't know what a miracle drug would be, but thisis about as close as any drug has come.

*

GEORGE DEMETRI: Go ahead and take a nice deep breath again. Again. Again.

GEORGE DEMETRI: So when we started doing the work on what now is known as Gleevec, we decided to look at an unusual kind of cancer called Gastro Intestinal Stromal Tumor - GIST, for short. For GIST patients, once this disease got beyond the control of the surgeon, 100% of people would die within a year and a half to two years.

GEORGE DEMETRI: First and most importantly, your scan looks great.

DOROTHY CLUKEY: Oh good.

GEORGE DEMETRI: So let's get rid of the any anxiety right off the top. I actually can show you the pictures if you would want to see the pictures of - that you just had. So the way these are done, so this is a picture...

GEORGE DEMETRI: We knew that cancer starts with one cell in a patient.

GEORGE DEMETRI: That cell makes a mutation; makes a mistake in copying the code of life: the DNA. Sometimes that gives rise to a funny-shaped protein that constantly signals to divide, to divide, to divide, to not stop, to keep going, to keep going, to keep going, and it doesn't know how to shut that off. It divides uncontrollably, it spreads from where it starts, and it kills the very person that gave birth to it.

GEORGE DEMETRI: At the top of the liver, we've been following this tiny little thing there, which is probably a little bit of dep - depositive tumor that the Gleevec is controlling beautifully. Now you're just taking one pill, right?

DOROTHY CLUKEY: No.

GEORGE DEMETRI: The 400 milligram pills? Or are you still getting the...

DOROTHY CLUKEY: Well, I'm still getting the, uh, it's 400...

GEORGE DEMETRI: And our team did some spectacular experiments in the laboratory to show that a molecular switch was indeed abnormally turned on in the cancer cells in GIST, and that you could shut off this switch inside the cancer cells with this new drug, now known as "Gleevec."

GEORGE DEMETRI: The Gleevec shuts off the enzymes, and...

DOROTHY CLUKEY: Oh.

GEORGE DEMETRI: ...the cells basically stop. They just get stuck and, actually, many of them were killed way back in the year 2000 when you started taking this.

DOROTHY CLUKEY: Mmm-hmm. Right. So this Gleevec is still working?

GEORGE DEMETRI: Yep.

DOROTHY CLUKEY: Wow.

GEORGE DEMETRI: It's turned into the standard of care.

NEWS REPORTER 3: This drug blocks the action of an enzyme that causes GIST, leaving the healthy cells untouched.

NEWS REPORTER 4: It does it by targeting proteins that signal cancer cells to multiply. Gleevec seems to block those signals. Without them the cancer cells die, fast. And with few side effects.

GEORGE DEMETRI: The excitement here goes well beyond Gleevec, into how we'll learn from this experience and move forward the whole field of drug development.

GEORGE DEMETRI: Every, every drug company's trying to get a drug approved always talks about unmet medical need. And this was a definite unmet medical need, because chemotherapy never worked.

GEORGE DEMETRI: As much as Gleevec was lifesaving and breakthrough and, and a revolution for the people with GIST, my email box was overflowing - I was still getting 600 emails a day saying, you know, "Could this help my mother? Could this help my loved one?" And it was really painful for me to say, "Look, you know, this was a relatively simple disease with one key mutation, and this drug targeted that mutation beautifully." But there are probably hundreds of mutations, maybe ten or twenty of which are really critical to driving the common cancers, like breast cancer, like lung cancer, like pancreatic cancer. So now in the modern era, we're really trying to find other cancer pathways, where we can basically take a molecular wrench, fix that short circuit, and not hurt the normal cells. And that's the whole concept behind smart drugs, designer drugs, to all these new drugs that are coming through pipelines and clinical trials that everybody's so excited about.